PepGen Inc. Announces Clinical Hold in the U.S. on IND Application to Initiate a Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)
31 May 2023
INDOligonucleotidePhase 1
PepGen Inc. Announces Clinical Hold in the U.S. on IND Application to Initiate a Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)
PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding their Investigational New Drug Application (IND) to initiate a Phase 1 study of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1 (DM1). The FDA indicated its intention to provide an official clinical hold letter to PepGen stating the reasons for the clinical hold within 30 days.
“We are disappointed to receive a clinical hold notice on our planned PGN-EDODM1 study in the U.S., and we will work closely with the FDA to lift the hold as quickly as possible,” said James McArthur, Ph.D., President and CEO of PepGen. “In parallel, we continue to pursue the advancement of PGN-EDODM1 into the clinic outside the U.S. We remain well-capitalized to fund the continued development of both EDO51 and EDODM1, investigational treatments that may have life-changing impact on individuals with neuromuscular disorders.”
As previously communicated, PepGen received a No Objection Letter (NOL) from Health Canada for its Clinical Trial application (CTA) to initiate the Phase 2 CONNECT1-EDO51 open label, multiple ascending dose (MAD) clinical trial of the Company’s lead asset PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) amenable to an exon 51 skipping approach. The clinical hold in the U.S. placed on PGN-EDODM1 does not impact the CONNECT1-EDO51 study which has been cleared to proceed in Canada.
About PepGen
PepGen Inc. is a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide, or EDO, platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates that are designed to target the root cause of serious diseases.
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